Not recruitingRare disease

Long-term safety and effectiveness study of leniolisib for patients with primary immunodeficiency and immune system problems

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study looks at a condition called primary immunodeficiency, which is a disorder where the immune system does not work properly and can lead to various health problems including abnormal immune responses in the body. The treatment being studied is leniolisib, which is also known by its code name CDZ173. This medication comes in the form of film coated tablets that are taken by mouth. The study will use different dose strengths of the medication, with maximum daily amounts of 20, 60, or 140 milligrams depending on what is appropriate for each person.

The purpose of this study is to assess the long-term safety and how well the body tolerates leniolisib when used to treat immune problems in people with primary immunodeficiency. This is an extension study, which means it is designed for people who have already participated in earlier studies of this medication and who their doctors believe would benefit from continuing to take leniolisib. The study will monitor for any unwanted effects or side effects through regular check-ups, vital sign measurements, safety laboratory tests, and physical examinations.

During the study, participants will continue taking leniolisib for an extended period, with the treatment lasting up to 156 weeks. The study will track various health measures over time, including blood cell counts such as hemoglobin, platelets, and different types of white blood cells. Doctors will also monitor lung function through breathing tests and imaging scans, check for changes in lymph nodes and spleen size, and measure certain substances in the blood that indicate how the immune system is functioning. All of these measurements help researchers understand how well the medication works over a long period and whether it remains safe for continued use.

The research process

The trial runs in 9 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Transition from previous study
Your participation in this extension study begins after completing a previous study called LE 7201 or LE 8201.
Your doctor will determine if continuing treatment with leniolisib would be beneficial for your condition.
Before starting, you or your legal representative (if you are under 18 years of age) will need to provide written informed consent. An interpreter will be available if needed.
Step 2
Ongoing treatment phase
You will receive leniolisib (also known as CDZ173) on a long-term basis throughout the study.
The medication is provided as film-coated tablets that are taken by mouth.
The specific dosage, frequency, and duration of treatment will be determined by your doctor based on your individual needs.
This is an open-label study, which means both you and your doctor will know that you are receiving leniolisib.
Step 3
Regular safety monitoring
Throughout the study, your safety will be continuously monitored through various assessments.
Your vital signs (such as blood pressure, heart rate, and temperature) will be measured regularly.
You will undergo physical examinations to check your overall health status.
Blood tests will be performed to monitor your safety and how your body is responding to the medication.
Any side effects or health changes you experience will be recorded and evaluated.
Step 4
Blood count monitoring
Regular blood tests will measure your hemoglobin levels (a protein in red blood cells that carries oxygen).
Your platelet count (cells that help blood clot) will be checked over time.
Your ANC (absolute neutrophil count, a type of white blood cell that fights infections) will be monitored regularly.
Step 5
Lung function assessment
You will have CT scans (detailed imaging tests) to examine your lungs for any changes related to your immune condition. These scans will look for granulomatous lymphocytic ILD (a type of lung inflammation) or other lung problems related to primary immunodeficiency (a condition where the immune system does not work properly).
Pulmonary function tests (breathing tests) will be performed to measure how well your lungs are working. These tests will measure several values including FEV1 (the amount of air you can forcefully exhale in one second), FVC (the total amount of air you can exhale), TLC (total lung capacity), RV (residual volume, or air remaining in lungs after exhaling), and DLCO (a measure of how well oxygen passes from the lungs into the blood).
Step 6
Lymph node and organ size monitoring
Measurements will be taken to assess any lymphoproliferation (abnormal growth of lymph tissue). This will be done by measuring the SPD (sum of the products of diameters) of specific lymph nodes that were identified at the start of your previous study.
Both index lesions (main areas of concern selected for tracking) and non-index lesions (other affected areas) will be measured over time.
Your spleen size will be monitored using both 3D volume (three-dimensional measurement) and 2D size (two-dimensional measurement) assessments.
Step 7
Immune cell monitoring
Blood tests will measure your CD4+ T cell count (a type of immune cell that helps coordinate the immune response).
Your CD8+ T cell count (immune cells that directly attack infected cells) will be monitored.
Your B cell count (immune cells that produce antibodies) will be checked regularly.
The percentages of naïve B cells (new B cells that have not yet encountered an antigen) and CD21low B cells (a specific type of B cell associated with immune dysfunction) will be measured over time.
Step 8
Immune marker monitoring
Blood tests will measure levels of CXCL13 (a protein that attracts certain immune cells) over time.
Levels of soluble IL-2Rα (a marker of immune system activation) will be monitored throughout the study.
Step 9
Long-term follow-up
This extension study is designed to evaluate the long-term safety and effectiveness of leniolisib for treating immune system problems in people with primary immunodeficiency.
The study is expected to continue until September 2029, allowing for extended observation of how the medication affects your condition over time.
All assessments and monitoring activities will continue at regular intervals throughout your participation in the study.

Who can join the trial?

4 criteria

You must have taken part in a previous study called LE 7201 or LE 8201
Your doctor believes that continuing treatment with leniolisib, which is the medicine being studied, will be helpful for you
You or your legal representative, if you are under 18 years old, must be able to talk with the study doctor and understand what the study requires
You or your legal representative must be able to sign a written agreement to join the study before any tests are done. An interpreter can help if you do not speak English

Who cannot join the trial?

3 criteria

The source data does not provide specific exclusion criteria for this clinical trial
Exclusion criteria are reasons why someone cannot join a study, such as having certain other medical conditions, taking specific medications, or having abnormal laboratory test results (blood tests or other medical tests done in a lab)
Without detailed information, it is not possible to list the specific reasons that would prevent participation in this particular study

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Verified sites

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Hospital Universitario Y Politecnico La Fe
Valencia, Spain

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Investigated drugs

Leniolisib is a medication being studied for treating immune system problems in patients with primary immunodeficiency. Primary immunodeficiency is a condition where the immune system does not work properly from birth. This medication aims to help control immune dysregulation, which means it tries to fix problems where the immune system is not working in a balanced way. In this study, researchers want to see if leniolisib is safe to use over a long period of time and if it continues to help patients with their immune system problems.

Leniolisib Phosphate

What is already known about the treatment

Leniolisib – Leniolisib is an oral medication taken by mouth that is being studied for the treatment of immune dysregulation in patients with primary immunodeficiency, a condition where the immune system does not work properly from birth. This drug works at the molecular level by blocking a specific protein called PI3K delta, which helps control how immune cells grow and function, thereby reducing abnormal immune system activity. Currently, leniolisib is undergoing clinical trials to evaluate its long-term safety, tolerability, and effectiveness in managing immune-related problems in people with primary immunodeficiency disorders. It belongs to a class of medications known as kinase inhibitors, which are drugs that interfere with specific enzymes involved in cell signaling.

Investigated diseases

Primary Immunodeficiency – Primary immunodeficiency is a group of disorders in which part of the body's immune system is missing or does not function properly from birth. These conditions are caused by genetic defects that affect the development or function of immune cells. People with primary immunodeficiency have increased susceptibility to infections that may be frequent, severe, or caused by unusual organisms. The disease can also lead to immune dysregulation, where the immune system attacks the body's own tissues or causes abnormal inflammation. Some forms may cause lymphoproliferation, which is an excessive growth of lymphoid tissue such as lymph nodes and spleen. Additional complications can include lung problems such as interstitial lung disease, low blood cell counts, and the formation of granulomas in various organs.